Benitec Biopharma(BNTC) - 2026 Q1 - Quarterly Report

Product Development - Benitec Biopharma is developing BB-301, a first-in-class genetic medicine for Oculopharyngeal Muscular Dystrophy (OPMD), utilizing a "silence and replace" approach[150]. - The Investigational New Drug (IND) application for BB-301 was approved by the U.S. FDA in June 2023, with the first subject treated in November 2023[152]. - BB-301 has been granted Orphan Drug Designation in both the United States and the European Union[150]. - The company aims to leverage its proprietary ddRNAi technology to achieve permanent silencing of disease-causing genes and restoration of normal gene function[140]. - The silence and replace approach allows for the simultaneous silencing of disease-causing genes and replacement with wild type genes, potentially improving treatment outcomes[130]. - BB-301 is designed to permanently silence the expression of the disease-causing gene while replacing it with a wild type gene[129]. - The BB-301 clinical development program includes a 76-week follow-up, with a 6-month pre-treatment observation period for OPMD patients[167]. - All six subjects in the BB-301 trial have been safely treated, with no treatment-related severe adverse events reported[169]. - Cohort 1 patients in the BB-301 trial achieved a 100% response rate, demonstrating significant improvements in dysphagia symptoms[170]. - Interim results showed a 40% decline in Total Pharyngeal Residue (TPR) and a 42% decline in Pharyngeal Area at Maximum Constriction (PhAMPC) post-treatment[175]. - The FDA granted Fast Track designation to BB-301 for the treatment of OPMD with dysphagia following positive interim clinical results[170]. Financial Performance - The company has not generated any revenues from product sales, with no revenue recognized during the three months ended September 30, 2025, and September 30, 2024[192]. - Total operating expenses for the three months ended September 30, 2025, were $9.8 million, a 69.5% increase from $5.8 million in the same period of 2024[196]. - Research and development expenses decreased to $3.4 million for the three months ended September 30, 2025, down from $3.6 million in the comparable period, primarily due to timing of contract manufacturing activities[196]. - General and administrative expenses rose significantly to $6.4 million in Q3 2025, compared to $2.2 million in Q3 2024, driven by a $4.0 million increase in share-based compensation[197]. - Other income (loss), net for the three months ended September 30, 2025, was $838 thousand, compared to $732 thousand in the same period of 2024, reflecting an increase in interest income[198]. - The company reported accumulated losses of $237 million as of September 30, 2025, with expectations of increased research and development expenses[200]. - Cash and cash equivalents stood at approximately $94.5 million as of September 30, 2025, with no borrowings or credit facilities in place[201]. - Net cash used in operating activities was $3.4 million for the three months ended September 30, 2025, an improvement from $4.6 million in the same period of 2024[203]. - The company has no products approved for sale and does not expect to generate significant revenue until regulatory approval is obtained[207]. - Future funding requirements will depend on various factors, including the timing and costs of clinical trials and regulatory approvals[209]. - The company anticipates continued losses for the foreseeable future as it develops product candidates and prepares for commercialization[208]. Strategic Collaborations and Financing - The company plans to collaborate with large biopharmaceutical companies for co-development and commercialization of its products[147]. - The company will require additional financing to progress its product candidates through key inflection points[131]. - The company is exploring long-term manufacturing alliances to support future commercialization efforts, as it does not currently own manufacturing facilities[177]. - The company entered into a Sales Agreement allowing for the sale of up to $75 million in common stock, with a 3.0% commission to the agent[190]. Market and Disease Context - OPMD is characterized by progressive swallowing difficulties and eyelid drooping, typically presenting in patients aged 40 to 50[149]. - Significant geographical clustering of OPMD patients has been noted, which could facilitate clinical development and commercialization efforts[158]. - BB-301 has received Orphan Drug Designation in the U.S. and EU, with a projected commercial opportunity exceeding $1 billion over its product life[160].